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Amyotrophic lateral sclerosis (ALS)
The purpose of this research is to determine if Nuedexta, an FDA approved and routinely prescribed medication, improves speech and swallowing functions in people with ALS.
This study will comprise an 18-week open-label safety and tolerability trial.
The investigators are interested in the therapeutic potential of acute intermittent hypoxia (AIH) for individuals with neuromuscular diseases, such as ALS.
The purpose of this study is to investigate the effects of two treatment techniques called Expiratory Muscle Strength Training (EMST) and Lung Volume Recruitment (LVR) on breathing, swallowing, speech, and cough function in persons with mild to moderate ALS.
The purpose of this study is to evaluate the discriminant ability of several non-invasive screening tools at detecting swallowing impairment in individuals with ALS.
This registry will ask participants diagnosed with ALS for clinical and demographic data to help researchers discover future treatments.
The purpose of this research study is to determine how accurate different tests are at identifying swallowing difficulties in persons with OPMD.
This study aims to help drug developers successfully design clinical trials and understand how to assess the efficacy of potential therapies. Researchers are trying to learn more about what causes the muscle weakness and stiffness (myotonia) in myotonic dystrophy.
This study is designed to obtain data regarding 2 aspects of the phenotype in myotonic dystrophy (dystrophia myotonica or DM). These are multi-system diseases leading to symptoms in many regions of the body including skeletal muscles, central nervous system and the GI tract.
This study is designed to assess social cognition deficits in adult onset patients with myotonic dystrophy type 1 and understand possible neuroimaging correlates of such deficits.
Study A083-02 is a multicenter, Phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE 083 in patients with FSHD to be conducted in two parts. Part 1 is open-label, dose-escalation and Part 2 is randomized, double-blind, and placebo-controlled.
The purpose of this research study is to evaluate a new investigational product called “SGT-001” which is designed to target the underlying cause of DMD in male children and adolescents (ages 4-17), by producing a shortened version of the dystrophin protein.
The main purpose of this study is to learn how well the investigational drug givinostat works and how safe givinostat is compared to placebo in males 6 years old to 17 years old.
We are doing this research study to find out if RO7239361 (previously known as BMS-986089) can help people with Duchenne.